The objectives of this study are to evaluate the efficacy of Valproic Acid (VPA) to both slow the progression of visual function loss and/or to restore visual function in patients with Autosomal Dominant Retinitis Pigmentosa (RP) and to collect safety and tolerability information.

One to four 250mg softgels by mouth daily (dose determined by body weight)

Other Name: Valproate

Placebo Comparator: Placebo

Subjects who receive placebo

Drug: Placebo

Dosage per subject weight- same schedule as the active comparator

Detailed Description:

Retinitis Pigmentosa (RP) is an incurable and untreatable group of heterogeneous retinal degenerative diseases that cause severe visual loss. There is currently no therapeutic that substantially slows the progression of this disease, and certainly none that can restore vision in RP patients. The Valproic Acid (VPA) study is designed as a six-site, interventional, prospective, randomized, placebo controlled, double-blinded study of 90 participants to evaluate the efficacy of oral Valproic Acid to both slow the progression of visual function loss and/or to restore visual function in patients with an Autosomal Dominant RP genetic mutation and to collect safety and tolerability information. Patients that participate in the study will be randomized to either placebo or VPA in a 1:1 ratio.

Visual acuity of greater than or equal to 35 letters in at least one eye as measured by the EVA-ETDRS (equivalent to 20/200 on a Snellen chart).

Genotyped as autosomal dominant form of RP.

Female subjects of childbearing potential and male subjects able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse or must commit to practice at least two acceptable methods of contraception to minimize the chance of pregnancy during the study and for the 13 week period after stopping the study drug.

Female subjects of childbearing potential must have a negative urine pregnancy test at study entry and throughout the duration of the study.

Willingness to comply with the protocol.

Exclusion Criteria:

Medical problems that make consistent follow-up over the treatment period unlikely (e.g. stroke, severe MI, end stage malignancy), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections.

Sensitive to or have ever had an allergic reaction to peanuts as peanut oil is an inactive ingredient in valproic acid capsules and the placebo.

Has taken one of the disallowed drugs at least 2 weeks prior to randomization.

Pregnant women.

Lactating mothers who are breast feeding their babies.

RP patients involved in other clinical trials within the last 3 months.

Require enrollment by consent of a legally authorized representative.

Persons who are unable to read are not allowed to consent for themselves or others to participate in this study.

The potential participant lives in the same household as a current participant in this protocol.

Contacts and Locations

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To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01233609